THE 6‐minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study
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Cites background from "THE 6‐minute walk test and other en..."
...Clinical features of DMD Large-scale population studies have outlined the natural disease progression in affected humans (Table 1) (Bushby and Connor, 2011; Henricson et al., 2013; Magri et al., 2011; McDonald et al., 2013a; McDonald et al., 2013b; Spurney et al., 2014)....
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...Between ages 5 and 8, symptoms are often stabilized or even slightly improved (known as the ‘honeymoon’ period) in the absence of any treatment (Bushby and Connor, 2011; McDonald et al., 2013a; McDonald et al., 2010)....
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Cites background or methods from "THE 6‐minute walk test and other en..."
...7 meters difference in 6MWD should be considered the minimal clinically important difference (MCID), and it is clinically relevant for nmDMD patients.(26) Although not statistically significant, patients treated with ataluren 40 mg/kg/day demonstrated a 31....
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...6MWD, whereas a >8-s stair climb predicts greater likelihood of loss of ambulation over 1 year.(26) The methods used by patients to perform these tests...
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...Completion of this trial has provided a better understanding of the natural history of DMD using the 6MWT and has established the 6MWT as a validated primary endpoint in DMD clinical trials; in addition, the data from this trial have helped to identify the best secondary endpoints in DMD trials and lay the clinical trial groundwork for future therapies for this disease.(26)...
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...and, like the 6MWT, are predictive of the time for a patient to become nonambulatory.(26) Natural history data from the Cooperative International Neuromus-...
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330 citations
References
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"THE 6‐minute walk test and other en..." refers background in this paper
...Muscle Nerve 48: 343–356, 2013 Duchenne muscular dystrophy (DMD) is a disabling and life-threatening X-linked genetic disorder caused by defects in the gene for dystrophin that results in a progressive loss of functional muscle fibers and weakness with stereotypic functional consequences affecting mobility, progressive musculoskeletal deformities, upper limb impairment, impaired airway clearance and ventilation, cardiomyopathy, and premature death.(1,2) The past several years have seen increased interest by biopharmaceutical companies in conducting ground-breaking research and development into novel treatment agents for DMD....
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1,085 citations
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997 citations
"THE 6‐minute walk test and other en..." refers background in this paper
...Muscle Nerve 48: 343–356, 2013 Duchenne muscular dystrophy (DMD) is a disabling and life-threatening X-linked genetic disorder caused by defects in the gene for dystrophin that results in a progressive loss of functional muscle fibers and weakness with stereotypic functional consequences affecting mobility, progressive musculoskeletal deformities, upper limb impairment, impaired airway clearance and ventilation, cardiomyopathy, and premature death.(1,2) The past several years have seen increased interest by biopharmaceutical companies in conducting ground-breaking research and development into novel treatment agents for DMD....
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